SG11201607738WA - Compositions and methods for enhanced gene expression in cone cells - Google Patents

Compositions and methods for enhanced gene expression in cone cells

Info

Publication number
SG11201607738WA
SG11201607738WA SG11201607738WA SG11201607738WA SG11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA
Authority
SG
Singapore
Prior art keywords
compositions
methods
gene expression
cone cells
enhanced gene
Prior art date
Application number
SG11201607738WA
Inventor
Thomas W Chalberg
Jay Neitz
Maureen Neitz
Original Assignee
Adverum Biotechnologies Inc
Univ Washington
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to US201461954330P priority Critical
Priority to US201562127185P priority
Application filed by Adverum Biotechnologies Inc, Univ Washington filed Critical Adverum Biotechnologies Inc
Priority to PCT/US2015/021087 priority patent/WO2015142941A1/en
Publication of SG11201607738WA publication Critical patent/SG11201607738WA/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses
    • C12N2750/00011MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA Viruses ssDNA Viruses
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/24Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/30Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/42Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
SG11201607738WA 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells SG11201607738WA (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
US201461954330P true 2014-03-17 2014-03-17
US201562127185P true 2015-03-02 2015-03-02
PCT/US2015/021087 WO2015142941A1 (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells

Publications (1)

Publication Number Publication Date
SG11201607738WA true SG11201607738WA (en) 2016-10-28

Family

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Family Applications (2)

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SG11201607738WA SG11201607738WA (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells
SG10201810150UA SG10201810150UA (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells

Family Applications After (1)

Application Number Title Priority Date Filing Date
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Country Status (10)

Country Link
US (2) US10000741B2 (en)
EP (1) EP3119798A4 (en)
JP (1) JP2017510296A (en)
KR (1) KR20160135754A (en)
CN (1) CN106414474A (en)
AU (1) AU2015231439A1 (en)
CA (1) CA2942776A1 (en)
IL (2) IL247756A (en)
SG (2) SG11201607738WA (en)
WO (1) WO2015142941A1 (en)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TW201828951A (en) 2012-05-15 2018-08-16 澳大利亞商艾佛蘭屈澳洲私營有限公司 Adeno-associated virus (aav) sflt-1 treatment of age-related macular degeneration (AMD)
SG11201607738WA (en) 2014-03-17 2016-10-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
EP3265178A4 (en) * 2015-03-02 2018-09-05 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3448437A1 (en) * 2016-04-29 2019-03-06 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
WO2018134168A1 (en) * 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
WO2018232149A1 (en) * 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders

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Also Published As

Publication number Publication date
EP3119798A4 (en) 2018-04-04
SG10201810150UA (en) 2018-12-28
AU2015231439A1 (en) 2016-10-06
CA2942776A1 (en) 2015-09-24
US20180320145A1 (en) 2018-11-08
US10000741B2 (en) 2018-06-19
KR20160135754A (en) 2016-11-28
JP2017510296A (en) 2017-04-13
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